Two infants in remission from leukaemia after CAR T-Cell therapy

pharmafile | January 26, 2017 | News story | Research and Development  

Researchers at the Great Ormond Street Hospital have published the successful treatment of two infants suffering from leukaemia by treatment with CAR T-cells. This form of treatment is a world’s first and involves the T cells from the immune system of a donor being harvested, engineered and then infused into a patient.

The two patients were infants of 11 and 16 months old who had undergone previous treatment with chemotherapy and bone marrow transplants. The CAR T-cell therapy then was their last option, but it is still, as yet, an experimental treatment and is not commercially available. The process involved genetically altering immune cells (T cells) so that they can target the cancer affecting the patient directly without damaging other cells, unlike chemotherapy.

The first case of the treatment being used was in 2015, in the case of Layla Richards. Layla, over one year on, is still in remission and has had no signs of recurrence. The method was approached due to the proximity of research and treatment departments in Great Ormond Street Hospital – allowing conversation to be struck between Waseem Qasim, a consultant immunologist and the leader of the tests, and French biotech firm Cellectis, based in the same hospital. A chance was taken on trying the treatment outside of the clinical trial environment and it paid off for those involved. This success was followed by one later treatment, and the results have been published. While the same treatment, UCART19, is being tested in Phase 1 trials.

It is a similar method of treatment to currently on-market immunotherapy, but differs in that current immunotherapies take the cells from the patient themselves and then engineer them. This is a lot more expensive and the great benefit of CAR T-cell therapy is that the treatment could potentially be stored and administered immediately.

The research was countered by the suggestion that, as both patients were treated with chemotherapy, the success cannot reliably be placed upon the CAR T-cells only. On top of this, there is the risk of graft versus host disease (GVHD) – where the body fights against the foreign cells introduced into the body. Researchers successfully resolved this issue by steroid treatment and a bone marrow transplant.

Ben Hargreaves

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